ROCKET PHARMACEUTICALS, INC. (RCKT)

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

You should read the following discussion and analysis of our financial condition and results of operations together with the consolidated financial statements and related notes that are included elsewhere in this Quarterly Report on Form 10-Q and our annual report on Form 10-K, filed on February 26, 2026, with the SEC.

Some of the statements contained in this discussion and analysis or set forth elsewhere in this quarterly report on Form 10-Q, including information with respect to our plans and strategy for our business, constitute forward looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended (the "Securities Act"), and Section 21E of the Securities Exchange Act of 1934, as amended (the "Exchange Act"). We have based these forward-looking statements on our current expectations and projections about future events. The following information and any forward-looking statements should be considered in light of factors discussed elsewhere in this quarterly report on Form 10-Q particularly including those risks identified in Part II, Item 1A“Risk Factors” and our other filings with the Securities and Exchange Commission (the "SEC").

Our actual results and timing of certain events may differ materially from the results discussed, projected, anticipated, or indicated in any forward-looking statements. We caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from the forward-looking statements contained in this quarterly report on Form 10-Q. Statements made herein are made as of the date of the filing of this Form 10-Q with the SEC and should not be relied upon as of any subsequent date. Even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in this quarterly report on Form 10-Q, they may not be predictive of results or developments in future periods. We disclaim any obligation, except as specifically required by law and the rules of the SEC, to publicly update or revise any such statements to reflect any change in our expectations or in events, conditions or circumstances on which any such statements may be based or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements.

We caution readers not to place undue reliance on any forward-looking statements made by us, which speak only as of the date they are made.

Business Highlights

During the second quarter of 2026, Rocket continued executing on its strategy to advance a focused portfolio of genetic medicines for inherited cardiovascular diseases, while progressing commercialization of its first approved product. The Company strengthened its balance sheet through the completed monetization of its Rare Pediatric Disease Priority Review Voucher (PRV), continued advancing its late-stage Danon disease program following resumption of dosing, progressed development across its cardiovascular pipeline, and continued commercial launch preparations for KRESLADI™.

Major developments during the quarter included:

•
Completion of the $180 million sale of the Company's Rare Pediatric Disease Priority Review Voucher (PRV), providing substantial non-dilutive capital to support the Company's strategic priorities.

•
Continued advancement of the RP-A501 Phase 2 pivotal Danon disease study following resolution of the FDA clinical hold and resumption of patient dosing.

•
Continued execution across the Company's cardiovascular genetic medicines portfolio, including advancement of the RP-A501 (Danon disease), RP-A601 (PKP2-ACM) and RP-A701 (BAG3-DCM) programs.

•
Continued commercial launch activities for KRESLADI™, including Qualified Treatment Center onboarding, manufacturing readiness, patient identification, reimbursement planning, and commercial infrastructure development.

The second quarter of 2026 reflected continued execution against the Company's strategic priorities. Management remained focused on advancing its lead cardiovascular genetic medicines programs, preparing for the commercial launch of KRESLADI™, strengthening the Company's financial position through completion of the PRV monetization transaction, and maintaining disciplined execution across clinical, regulatory, manufacturing, and commercial activities.

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Overview

Rocket Pharmaceuticals is a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and devastating diseases, with a strategic focus on inherited cardiovascular conditions. Our prioritized development portfolio includes AAV-based gene therapies targeting genetically defined cardiomyopathies, complemented by KRESLADI™, our first FDA-approved product, for the treatment of pediatric patients with severe LAD-I who have biallelic mutations in the ITGB2 gene and do not have a suitable HLA-matched sibling donor. Our capabilities span clinical development, regulatory execution, manufacturing and commercialization, supported by in-house research and development expertise and AAV cGMP manufacturing infrastructure.

The Company’s activities during the quarter reflect continued execution across our prioritized cardiovascular genetic medicines programs, alongside commercial readiness activities for KRESLADI™. Given the ultra-rare patient population and anticipated phased commercial rollout, the Company does not expect KRESLADI™ to generate material revenue in the near term.

We aim to develop and commercialize genetic medicines that address the underlying causes of rare and devastating diseases with significant unmet need. Our current development strategy is centered on inherited cardiovascular diseases, where our scientific, clinical, manufacturing and regulatory capabilities may support a portfolio of differentiated and potentially first- or best-in-class therapies.

In July 2025, we announced a strategic corporate reorganization and pipeline prioritization initiative designed to maximize near-term value creation, extend our operational runway, and position the Company for sustainable long-term growth. The initiative concentrated development resources on advancing our AAV-based cardiovascular genetic medicines portfolio and supporting the submission of our response to the FDA’s CRL for KRESLADI™. As part of this strategic realignment, we de-prioritized further development activities related to our FA and PKD programs and implemented a workforce reduction of approximately 30%.

In March 2026, KRESLADI™ (marnetegragene autotemcel) received accelerated approval from the FDA for the treatment of pediatric patients with severe LAD-I who have biallelic mutations in the ITGB2 gene and do not have a suitable HLA-matched sibling donor. In connection with the approval, the Company was awarded a PRV and, in April 2026, entered into a definitive agreement to sell the PRV for $180 million. The transaction closed in June 2026. The Company intends to pursue a focused commercial strategy for KRESLADI™ that is appropriately scaled to the exceptionally small patient population affected by this ultra-rare disease.

Our strategy is built on several foundational pillars:

•
First-and-Best-in-Class Approach: With our program selection, we apply a rigorous, disease-based selection approach to identify and prioritize programs: targeting complex genetic disorders with differentiated therapies that offer the potential to be first-, best-, or only-in-class, focusing on monogenic disease with on-target mechanisms of action to directly address the root cause of the disease to offer superior clinical profiles, and choosing indications with sizable market opportunities to enable broad patient impact and sustainable value creation.

•
Strategic Focus on Rare Cardiovascular Indications: Our near-term research and development investments are focused on applying our AAV capabilities to genetically defined cardiovascular diseases. Collectively, our clinical cardiovascular genetic medicines programs address genetically defined forms of hypertrophic, arrhythmogenic and dilated cardiomyopathy, representing three major categories of inherited heart disease with significant unmet need.

•
Late-Stage Science & Innovation with Robust Capabilities: We are advancing promising clinical programs designed to support regulatory approvals in the U.S. and Europe, with potential expansion into Asia and beyond. To support our clinical and future commercial endeavors, we are currently operating a ~100,000 sq. ft. U.S.-based in-house AAV cGMP manufacturing facility in Cranbury, New Jersey.

•
Expertise & Collaboration: Our leadership team brings a proven track record of over 20 successful U.S. and international drug approvals and launches with expertise in cell and gene therapies and rare diseases. We collaborate closely with scientific experts, healthcare providers, payors, and patient communities to ensure our therapies address real-world needs.

In the near- and medium-term, we are focused on:

•
Advancing our portfolio of product candidates targeting monogenic cardiovascular diseases with substantial unmet need across stages of clinical development.

•
Continuing to build and scale proprietary in-house analytics, process development, and manufacturing capabilities to support clinical and commercial supply.

•
Evaluating potential strategic partnerships or other transactions for certain non-core programs to enable continued development, regulatory approval, and commercialization.

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In the medium- and long-term, pending favorable data, we plan to:

•
Submit BLAs for certain of our clinical programs.

•
Evaluate opportunities to expand our cardiovascular genetic medicines portfolio into additional genetically defined indications that are compatible with our AAV capabilities and core strategy.

•
Pursue potential eligibility for FDA priority review voucher programs.

Genetic Medicines Overview

Genetic medicines are a therapeutic approach in which an isolated gene sequence or segment of DNA is administered to a patient, most commonly for the purpose of treating a genetic disease that is caused by genetic mutations. Currently available therapies for many genetic diseases focus on administration of large proteins or enzymes and typically address only the symptoms of the disease. Genetic medicines aim to address the disease-causing effects of absent or dysfunctional genes by delivering functional copies of the gene sequence directly into the patient’s cells, offering the potential for curing the genetic disease, rather than simply addressing symptoms.

We are developing genetic medicine product candidates utilizing modified, non-pathogenic viruses as delivery vehicles. Viruses are inherently effective for gene delivery due to their natural ability to enter cells and deliver genetic material. In engineering our viral vectors, the native viral genes are removed and replaced with a functional copy of the missing or mutated gene responsible for a patient’s genetic disorder. This functional copy, known as the therapeutic gene or “transgene,” is introduced through a process known as transduction. Once modified, the virus is termed a “viral vector,” capable of delivering the transgene to targeted tissues or organs.

We are advancing genetic medicine programs using two primary vector approaches: adeno-associated virus (AAV) vectors and lentiviral (LV) vectors. We believe our AAV- and LV-based programs have the potential to provide meaningful and durable therapeutic benefit by addressing the underlying genetic cause of disease. Our genetic medicine product candidates are administered either (1) in vivo

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Item 7. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our consolidated financial statements, related notes and other financial information included elsewhere in this Annual Report. This discussion contains forward-looking statements that involve risks and uncertainties such as our plans, objectives, expectations, and intentions. Our actual results could differ materially from those discussed in these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those identified below and those discussed in “Risk Factors” included elsewhere in this Annual Report.

Unless otherwise indicated, references to “Rocket,” the “Company,” “we,” “our” and “us” refer to Rocket Pharmaceuticals, Inc. and its subsidiaries.

Introduction

We are a fully integrated, late-stage biotechnology company focused on the development, manufacturing, and potential commercialization of genetic therapies for rare and often fatal diseases with a high unmet medical need. Our innovative multi-platform approach allows for the creation of best-in-class gene therapy product candidates aimed at correcting the root cause of complex genetic disorders, spanning across cardiac and hematologic indications, offering the potential for transformative and durable clinical benefits. Rocket’s platform is supported by in-house R&D capabilities and current cGMP facilities that enable end-to-end control over clinical production and scale-up for commercialization.

We seek to bring hope and relief to patients with devastating, undertreated and rare diseases through the development and commercialization of potentially curative first-in-class gene therapies. As a fully integrated, late-stage biotechnology company, we have the resources and opportunity to generate a portfolio of highly differentiated and potentially first-in-class or best-in-class genetic medicines.

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In July 2025, we announced a strategic corporate reorganization and pipeline prioritization designed to maximize near-term value, extend our operational runway, and position the Company for sustained long-term growth. This initiative focuses our resources on advancing our AAV cardiovascular gene therapy platform and supporting the submission of our responses to the FDA’s CRL for KRESLADI™. The program contemplates a scaled commercial effort tailored to the exceptionally small patient population affected by this ultra-rare indication. As part of this strategic realignment, we are also de-prioritizing further development activities related to our FA and PKD programs. As part of the restructuring, the Company implemented a reduction in the workforce of approximately 30%, which, along with other planned cost-saving initiatives, is expected to reduce Rocket’s 12-month operating expenses by approximately 25%.

Our strategy is built on several foundational pillars:

•
First-and-Best-in-Class Approach: With our program selection, we apply a rigorous, disease-based selection approach to identify and prioritize programs: targeting complex genetic disorders with differentiated therapies that offer the potential to be first-, best-, or only-in-class, focusing on monogenic disease with on-target mechanisms of action to directly address the root cause of the disease to offer superior clinical profiles, and choosing indications with sizable market opportunities to enable broad patient impact and sustainable value creation.

•
Strategic Focus on Rare Cardiovascular Indications: Our near-term research and platform investments are focused on leveraging our AAV capabilities in rare cardiovascular diseases. Collectively, our clinical cardiovascular gene therapy programs target the major genetically defined causes of hypertrophic, arrhythmogenic, and dilated cardiomyopathies which represent a significant portion of inherited heart disease and impact more than 100,000 patients in the U.S. and EU.

•
Late-Stage Science & Innovation with Robust Capabilities: We are advancing promising clinical programs designed to support regulatory approvals in the U.S. and Europe, with potential expansion into Asia and beyond. To support our clinical and future commercial endeavors, we are currently operating a ~100,000 sq. ft. U.S.-based in-house AAV cGMP manufacturing facility in Cranbury, New Jersey.

•
Expertise & Collaboration: Our leadership team brings a proven track record of over 20 successful U.S. and international drug approvals and launches with expertise in cell and gene therapies and rare diseases. We collaborate closely with scientific experts, healthcare providers, payors, and patient communities to ensure our therapies address real-world needs.

In the near- and medium-term, we are focused on:

•
Advancing our first-in-class product candidates targeting monogenic diseases with substantial unmet need.

•
Building proprietary in-house analytics and manufacturing capabilities.

•
Conducting registration trials for our lead programs.

In the medium- and long-term, pending favorable data, we plan to:

•
Submit BLAs for certain of our clinical programs.

•
Expand our gene therapy platform to additional indications compatible with our technologies.

•
Pursue potential eligibility for FDA priority review vouchers, pending program renewal by Congress.

Financial Overview

Since our inception, we have devoted substantially all of our resources to organizing and staffing the Company, business planning, raising capital, acquiring, or discovering product candidates and securing related intellectual property rights, conducting discovery, R&D activities for our product candidates and planning for potential commercialization. We do not have any products approved for sale and have not generated any revenue from product sales. From inception through December 31, 2025, we raised net cash proceeds of approximately $1.2 billion from investors through both equity and convertible debt financing to fund operating activities.

Revenue

To date, we have not generated any revenue from any sources, including from product sales, and we do not expect to generate any revenue from the sale of products in the near future. If our development efforts for product candidates are successful and result in regulatory approval or license agreements with third parties, we may generate revenue in the future from product sales.

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Research and Development Expenses

Our R&D program expenses consist of both internal and external costs incurred for the development of our product candidates. These expenses include:

•
expenses incurred under agreements with research institutions and consultants that conduct R&D activities, including process development and preclinical and clinical activities on our behalf;

•
costs related to process development and production of preclinical and clinical materials, including fees paid to contract manufacturers and manufacturing input costs for use in internal manufacturing processes;

•
consultants supporting process development and regulatory activities; and

•
costs related to in-licensing of rights to develop and commercialize our product candidate portfolio.

We recognize external development costs based on contractual payment schedules aligned with program activities, invoices for work incurred, and milestones that correspond with costs incurred by the third parties. Non-refundable advance payments for goods or services to be received in the future for use in R&D activities are recorded as prepaid expenses.

Our direct R&D expenses are tracked on a program-by-program basis for product candidates and consist primarily of external costs, such as research collaborations and third-party manufacturing agreements associated with our preclinical research, process development, manufacturing, and clinical development activities. Our direct R&D expenses by program also include fees incurred under license agreements. Our personnel, non-program and unallocated program expenses include costs associated with activities performed by our internal R&D organization and generally benefit multiple programs. These costs are not separately allocated by product candidate and consist primarily of:

•
salaries and personnel-related costs, including benefits, travel, and stock-based compensation, for our scientific personnel performing R&D activities;

•
facilities and other expenses, which include expenses for rent and maintenance of facilities, and depreciation expense; and

•
laboratory supplies and equipment used for internal R&D activities.

We allocate salary and benefit costs directly related to specific programs. We do not allocate personnel-related discretionary bonus or stock-based compensation costs, costs associated with our general discovery platform improvements, depreciation or other indirect costs that are deployed across multiple projects under development and, as such, the costs are separately classified as other R&D expenses.

The following table presents R&D expenses, in thousands, tracked on a program-by-program basis as well as by type and nature of our expense for our product candidates for the years ended December 31, 2025 and 2024.

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We cannot determine with certainty the duration and costs to complete current or future clinical studies of product candidates or if, when, or to what extent we will generate revenues from the commercialization and sale of any of our product candidates that obtain regulatory approval. We may never succeed in achieving regulatory approval for any of our product candidates. The duration, costs, and timing of clinical studies and development of product candidates will depend on a variety of factors, including:

•
the scope, rate of progress, and expense of ongoing clinical studies as well as any clinical studies and other R&D activities that we undertake in the future;

•
future clinical study results;

•
uncertainties in clinical study enrollment rates;

•
changing standards for regulatory approval; and

•
the timing and receipt of any regulatory approvals.

We expect R&D expenses to increase for the foreseeable future as we continue to invest in R&D activities related to developing product candidates, including investments in manufacturing, as our programs advance into later stages of development and as we conduct additional clinical trials. The process of conducting the necessary clinical research to obtain regulatory approval is costly and time-consuming, and the successful development of product candidates is highly uncertain. As a result, we are unable to determine the duration and completion costs of R&D projects or when and to what extent we will generate revenue from the commercialization and sale of any of our product candidates.

Our future R&D expenses will

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